Enasidenib
Enasidenib is a targeted therapy medication used to treat acute myeloid leukemia (AML) in patients who have specific mutations in the IDH2 gene. It works by inhibiting mutant forms of the IDH2 enzyme, which helps restore normal blood cell development and reduce cancer cell growth. This medication is typically used in patients whose AML has returned or has not responded to previous treatments.
Enasidenib is a targeted therapy for AML patients with IDH2 mutations, working by inhibiting mutant IDH2 enzymes to help restore normal blood cell production.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- L01XM01
- RxNorm
- 1940332
- Generic
- Enasidenib
- Class
- atc-l01
- Brands
- Idhifa
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- tablet
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Enasidenib is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) who have specific IDH2 mutations (R140Q, R172S, R172K). Genetic testing is required to confirm the presence of these mutations before starting treatment.
Enasidenib is a small molecule inhibitor that specifically targets and blocks mutant forms of the isocitrate dehydrogenase 2 (IDH2) enzyme. By inhibiting these mutant IDH2 variants, it prevents the accumulation of an oncometabolite called 2-hydroxyglutarate (2-HG), which promotes abnormal blood cell development. This inhibition allows for normal differentiation of blood cells and reduces the proliferation of leukemia cells.
Common side effects may include nausea, vomiting, diarrhea, decreased appetite, and changes in blood counts. A serious but uncommon side effect is differentiation syndrome, which requires prompt medical attention if symptoms such as fever, difficulty breathing, or rapid weight gain occur. Regular monitoring of blood counts and liver function is recommended during treatment.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy